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Article

Allele-specific RNA interference for neurological disease.

Gene therapy - 1 Mar 2006

Rodriguez-Lebron E, Paulson H L

Abstract excerpt

Suppressing the expression of toxic genes through RNAi holds great promise for the treatment of human disease. Allele-specific approaches have now been used to silence dominant toxic genes implicated in several neurological disorders. Here, we review strategies used to achieve allele-specific silencing in light of recent developments in the field of RNAi biology. In particular, new insights into siRNA and miRNA...

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Identifiers

PMID: 16355113
DOI: 10.1038/sj.gt.3302702

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